FDA en­dors­es No­vavax's piv­otal tri­al de­sign for flu vac­cine; Forty Sev­en bags fund­ing for myelodys­plas­tic syn­dromes pro­gram

→ Fol­low­ing a stream of set­backs with its RSV vac­cine, No­vavax is tout­ing some reg­u­la­to­ry progress of an­oth­er can­di­date for a change. The FDA has ap­par­ent­ly green-light­ed the Phase III tri­al de­sign for NanoFlu, its ad­ju­vant­ed, re­com­bi­nant quadri­va­lent flu vac­cine tar­get­ing the el­der­ly (65 or old­er). The tri­al is ex­pect­ed to be­gin in a cou­ple of months and gen­er­ate top-line da­ta in the first quar­ter of 2020.

→ The an­ti-CD47 work that Forty Sev­en has been do­ing in myelodys­plas­tic syn­dromes has caught the at­ten­tion of The Leukemia & Lym­phoma So­ci­ety, which now wants to help ac­cel­er­ate the de­vel­op­ment of the ear­ly-stage 5F9 pro­gram. “De­spite in­cred­i­ble ad­vance­ments for pa­tients with oth­er hema­to­log­i­cal ma­lig­nan­cies, the treat­ment land­scape in MDS has re­mained un­changed for many years, with no new FDA-ap­proved ther­a­pies since 2006,” LLS CSO Lee Green­berg­er said in a state­ment. So far, the biotech — found­ed by Stan­ford’s Irv Weiss­man — has demon­strat­ed an im­pres­sive over­all re­sponse rate of 100%, more than half of which were com­plete re­spons­es. LLS is of­fer­ing $6 mil­lion to fund the up­com­ing tri­al and $3 mil­lion more for mile­stones, eye­ing a BLA fil­ing as ear­ly as 2021.

Leap Ther­a­peu­tics$LP­TX an­ti-DKK1 an­ti­body, DKN-01, in tan­dem with Mer­ck‘s $MRK flag­ship check­point in­hibitor Keytru­da in a study im­proved out­comes in pa­tients with gas­troe­sophageal junc­tion and gas­tric can­cer whose tu­mors are DKK1-high and who had not pre­vi­ous­ly been treat­ed with PD-1/PD-L1 im­munother­a­pies. “Out­side of rare mi­crosatel­lite in­sta­ble tu­mors and EBV-as­so­ci­at­ed can­cers the re­sponse rates to im­mune check­point in­hibitors are low and me­di­an pro­gres­sion-free sur­vival re­mains short, in the range of 6-8 weeks,” said Samuel Klemp­n­er, as­sis­tant pro­fes­sor, Mass­a­chu­setts Gen­er­al Hos­pi­tal Can­cer Cen­ter and Har­vard Med­ical School in a state­ment. “This study…sug­gests that el­e­vat­ed DKK-1 ex­pres­sion is a po­ten­tial pre­dic­tive bio­mark­er. DKN-01 war­rants fur­ther study in gas­troe­sophageal can­cers in com­bi­na­tion with im­mune check­point in­hibitors and with chemother­a­py.”

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can created herd immunity in the US by the middle of next year, with Europe following a few months later.

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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.

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.

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.

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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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.

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.