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.

Zo­genix plans quick re­turn to the FDA with their spurned ap­pli­ca­tion on Dravet syn­drome drug — shares spike

Zo­genix shares are claw­ing back some of the val­ue they lost 2 months ago af­ter the FDA hit the biotech with a refuse-to-file no­tice on their ex­per­i­men­tal ther­a­py for Dravet syn­drome. 

Com­pa­ny ex­ecs said this morn­ing that they worked out reg­u­la­tors’  is­sues with the ap­pli­ca­tion for Fin­tepla, which cen­tered on a pair of big prob­lems: the ab­sence of non-clin­i­cal stud­ies need­ed to al­low as­sess­ment of the chron­ic ad­min­is­tra­tion of fen­flu­ramine and the in­clu­sion of an in­cor­rect ver­sion of a clin­i­cal dataset. Now they plan to re­sub­mit in Q3 af­ter get­ting off the hook on both scores — which trig­gered a sigh of re­lief among in­vestors.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

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As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

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Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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