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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.