Pharnext rare dis­ease drug scores in late-stage study, tee­ing up ap­pli­ca­tions at the FDA and EMA

French drug de­vel­op­er Pharnext SAS says their cru­cial late-stage study has come through with sig­nif­i­cant re­sults for a new drug to ad­dress a rare neu­ro­log­i­cal dis­ease that has so far elud­ed treat­ment. The com­pa­ny’s stock (EPA: $AL­PHA) soared 25% Tues­day morn­ing af­ter it an­nounced that the ex­per­i­men­tal treat­ment for Char­cot-Marie-Tooth (CMT) dis­ease was com­pelling enough to ap­ply for FDA and EMA ap­proval.

CMT is an in­her­it­ed dis­ease caused by gene mu­ta­tions that re­sult in pe­riph­er­al nerve dam­age, and is char­ac­ter­ized by the pro­gres­sive mus­cle wast­ing of the ex­trem­i­ties as well as sen­so­ry loss. Cur­rent­ly, pa­tients are treat­ed with or­tho­pe­dic de­vices, phys­i­cal ther­a­py and pain killers. There ex­ist no treat­ments that di­rect­ly tar­get the dis­ease, and the most com­mon 1A form of the dis­ease af­fects at least 125,000 peo­ple in the US and EU.

The ex­per­i­men­tal treat­ment, PXT-3003, is com­prised of three ap­proved drugs — ba­clofen, nal­trex­one and sor­bitol. In the Phase III tri­al, two dos­es of the drug were test­ed against a place­bo in pa­tients with mild-to-mod­er­ate CMT type 1A over a 15-month pe­ri­od. The main goal of the study, which en­rolled 323 pa­tients aged 16 to 65 years, was to get at least a 0.3 re­duc­tion in a scale mea­sur­ing pa­tient dis­abil­i­ty called Over­all Neu­ropa­thy Lim­i­ta­tion Scale (ONLS).

Daniel Co­hen

Da­ta showed a mean re­duc­tion of 0.4-point on the ONLS (95% CI [0.1,0.6], p=0.008) in the high­er dose group com­pared to the place­bo. Sec­ondary end­points con­firmed the su­pe­ri­or­i­ty of the high­er dose, in par­tic­u­lar demon­strat­ing im­prove­ment on a 10-me­ter walk test with a re­duc­tion of 0.5 sec (95%CI [0.1,0.9], p=0.016), Pharnext added.

In the tri­al, 87 pa­tients were giv­en a place­bo, 93 pa­tients were giv­en the low­er dose and 55 pa­tients got the high­er dose. The low­er num­ber of pa­tients giv­en the high­er dose is due to un­ex­pect­ed for­mu­la­tion/sta­bil­i­ty is­sues, the com­pa­ny said, adding that the treat­ment was found to be safe and well tol­er­at­ed.

Un­der the stew­ard­ship of its CEO Daniel Co­hen, who was one of the re­searchers in­stru­men­tal in map­ping the hu­man genome, Pharnext has de­vel­oped its tech­nol­o­gy based on a sim­i­lar mod­el. Its pleother­a­py plat­form iden­ti­fies the ide­al com­bi­na­tion of avail­able med­i­cines to fight tar­gets un­der­ly­ing dis­ease.

Pharnext plans to file for mar­ket­ing ap­proval first with the FDA, Co­hen told End­points News, and ex­pects the drug will of­fi­cial­ly hit the mar­ket by 2020, but in­tends to make the drug avail­able to pa­tients soon­er via ear­ly-ac­cess pro­grams. His ap­proach to pric­ing is al­so strik­ing­ly dif­fer­ent.

“Af­ter many dis­cus­sions with pay­ers, we’re think­ing of pric­ing it at 10,000 eu­ros in Eu­rope, and around 40,000 – 50,000 dol­lars in the US, as sug­gest­ed by the pay­ers them­selves,” he said.

Co­hen has long sug­gest­ed that he is against the stan­dard in­dus­try ap­proach to rare drug pric­ing, in which treat­ments for rare dis­eases typ­i­cal­ly ex­ceed $100,000 per pa­tient per year.

PXT-3003, which has pro­cured or­phan drug des­ig­na­tions in the US, EU and Chi­na, is al­so be­ing eval­u­at­ed in an on­go­ing fol­low-up ex­ten­sion study that’s ex­pect­ed to pro­duce re­sults in the sec­ond half of next year, and a pe­di­atric study is set to com­mence in the first half of 2019. Mean­while, Pharnext’s sec­ond drug in de­vel­op­ment, PXT864, is be­ing eval­u­at­ed as a treat­ment for Alzheimer’s and Lou Gehrig’s dis­ease.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Days af­ter In­ter­cept re­jec­tion, Akero surges on ‘un­prece­dent­ed‘ NASH da­ta

A year and a half after scoring a $70 million Series B and a top Gilead executive as CEO, Akero Therapeutics has announced new data on their NASH drug. And with the field still reeling from a surprise FDA rejection this week, the news was enough to send their stock surging.

Akero had already said in March that its lead drug had beaten placebo in its Phase II trial, reducing liver fat by 14% in the highest dose group compared to 0.3% in placebo, according to MRI scans. But although NASH is an obesity-related condition and results from fatty buildup in the liver, the real immediate question for any therapy is whether it can resolve the fibrosis and inflammation that results from that buildup. Those data require biopsying the patients, a longer and more invasive process that was further complicated by a pandemic.